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Table 3 Safety information before miglustat initiation and during NPC Registry follow up

From: Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study

Safety information/event Time period Patients, N Patients with events, n (%)**
Seizures Pre-treatment: present 90 21 (23)
  During follow-up: worsened* 39 13 (33)
  During follow-up: new* 39 13 (33)
Thrombocytopenia Pre-treatment: present 90 18 (20)
  During follow-up: present* 80 43 (54)
Tremor Pre-treatment: present 91 23 (25)
  During follow-up: worsened* 91 6 (7)
  During follow-up: new* 91 2 (2)
Neuropathy Pre-treatment: present 90 4 (4)
  During follow-up: worsened* 91 7 (8)
  During follow-up: new* 91 1 (1)
Chronic diarrhea Pre-treatment: present 91 2 (2)
  During follow-up: present* 92 10 (11)
Other†† During follow-up: present* 92 4 (4)
  1. *Event occurred at least once during follow-up; **percentages calculated according to number of patients with available data; 38 mild thrombocytopenia (101–150 × 109/L) and 10 moderate thrombocytopenia (51–100 × 109/L); diarrhea lasting >3 months; ††other adverse events comprised: dental cavities, loss of consciousness and circulatory collapse, chronic diarrhea and new seizures (all n = 1)